Our Innovative Treatments and Therapies

Through the CRDT patients with rare diseases have access to innovative therapies and research including:

  • Liver transplantation as a cure or treatment for metabolic disease, having transplanted more patients with metabolic disease than any other center
  • Treatment of 10 to 12 non-malignant patients per year with stem cell transplants resulting in a<5 percent mortality at 100 days
  • Innovative surgical techniques such as cutting-edge corneal transplants
  • One of a few centers in the world offering total pancreatectomy with islet cell autotransplant for children with chronic pancreatitis
  • Utilizing reduced-toxicity/intensity in unrelated donor, cord blood transplantation• Exceptional medical management of metabolic patients leading to better outcomes
  • Approved and experimental enzyme replacement therapies for inborn errors of metabolism 
  • FDA-approved clinical trials for novel therapies for fatty acid oxidation disorders 
  • Hepatocyte transplantation therapy for inborn errors of metabolism including urea cycle disorders and PKU, and acute hepatic failure in patients with metabolic disorders 
  • NIH-funded study to test the possibility that the commonly used mood-stabilizer, Carbamazepine, can reverse liver disease due to alpha-1-antitrypsin deficiency 
  • FDA-approved study of sequential lung and bone marrow transplantation from the same cadaveric donor 
  • Groundbreaking transplant research, including advances in immunosuppression withdrawal or minimization, transplant tolerance, and optimizing long-term transplant outcomes